FDA opens up approval process for RYONCYL™

Mesoblast Ltd, headquartered in Australia with subsidiaries in the USA and Singapore, develops and distributes innovative allogenic cell therapeutics to treat complex diseases for which conventional therapies fail or are not available. The company focuses on the anti-inflammatory and regenerative potential of mesenchymal stromal cells (MSC) and their progenitors (MPC).

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On August 13, 2020 Mesoblast achieved a major breakthrough: the United States Food and Drug Adminsitration (FDA) recognized the efficacy of the ATMP RYONCIL™ (remestemcel-L) in children with steroid-refractory acute graft versus host disease (GvHD) and included it in the priority review after the ODAC (Oncologic Drugs Advisory Committee) issued a recommendation. The next steps require FDA approval of the drug under the Prescription Drug User Fee Act (PDUFA) by the end of September. Then RYONCIL™ can be launched on the US market not later than end of 2020.

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Nine clinical studies have been conducted so far, most of them under the name of the precursor product Prochymal®. The last phase III study was recently completed. In Canada, New Zealand and Japan (TEMCELL®HS Inj.), this cell preparation has already received approval some years ago and has so far been used mainly in children who developed severe GvHD after stem cell transplantation and who could not be treated with the conventional treatment procedures.

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Now this therapy will also be available in the US.

Author: Dr. Annette Hogardt-Noll, GMP Compliance Services for ATMPs, Valicare GmbH, Frankfurt/Germany